Investigation of Cognitive Outcomes With Cannabidiol Oral Solution

NCT04133480 | Withdrawn Phase 4 FDA Drug

• 1 other identifier: GWEP18060
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study is being conducted to evaluate the effects of GWP42003-P on cognition in pediatric participants, aged 3 to 10 years, with Lennox-Gastaut Syndrome (LGS).

Conditions

Lennox-Gastaut Syndrome

Keywords

GWP42003-P; EPIDIOLEX®; cannabidiol; CBD; cognition

Outcome Measures

Primary Outcomes (1)

• Change from Baseline to end of treatment (Day 181 [Visit 5]) in processing speed on the National Institutes of Health Toolbox Cognition Battery (NIHTCB)

  Baseline; Day 181

Secondary Outcomes (20)

• Change from Baseline to Day 91 (Visit 4) in processing speed on the NIHTCB

  Baseline; Day 91

• Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in executive function and attention on the NIHTCB

  Baseline; Days 91 and 181

• Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in episodic memory on the NIHTCB

  Baseline; Days 91 and 181

• Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in language on the NIHTCB

  Baseline; Days 91 and 181

• Change from Baseline to Day 91 (Visit 4) and Day 181 (end of treatment; Visit 5) in the NIHTCB Childhood Composite Score

  Baseline; Days 91 and 181

Study Arms (1)

GWP42003-P

EXPERIMENTAL

For the first 7 days of the treatment period, participants are to take GWP42003-P at a dose of 5 milligrams per kilogram per day (mg/kg/day), administered as 2 equally divided doses (i.e., 2.5 mg/kg in the morning and 2.5 mg/kg in the evening). On Day 8, participants are to increase the dose to 10 mg/kg/day, administered as 2 equally divided doses (i.e., 5 mg/kg in the morning and 5 mg/kg in the evening). The 10 mg/kg/day dose should be maintained for the remainder of the treatment period; however, per labeling, investigators may increase the dose to a maximum of 20 mg/kg/day if clinically warranted by titrating an additional 5 mg/kg/day each week until reaching the maximum dose. GWP42003-P will be taken b.i.d. (morning and evening).

Drug: GWP42003-P

Interventions

GWP42003-P DRUG

oral solution of 100 milligrams per milliliter (mg/mL) cannabidiol (CBD)

Also known as: Cannabidiol, CBD, Epidiolex

GWP42003-P

Eligibility Criteria

• Age: 3 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant is male or female aged 3-10 years.
• Participants' parent(s)/legal representative is willing and able to give informed consent for participation in the trial; where the participant possesses adequate understanding, informed assent should also be taken.
• Participant and their caregiver are willing and able (in the investigator's opinion) to comply with all trial requirements.
• Participant must have a clinical diagnosis of Lennox-Gastaut Syndrome (LGS), with onset within the last 5 years. This includes certification from the investigator of prior electroencephalogram (EEG) documenting slow spike wave (\< 3 Hertz \[Hz\]) during the participant's history and evidence of more than 1 type of generalized seizure, including drop seizures (atonic, tonic, or tonic-clonic), for at least 6 months.
• Investigator can confirm that the addition of GWP42003-P to the participant's existing antiepileptic drug (AED) regimen is warranted.
• Participant must have at least 1 drop seizure each week during the first 28 days of the baseline period.
• A minimum level of general intellectual functioning as assessed at screening with the Peabody Picture Vocabulary Test
• Participant's parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
• Participant's parent(s)/legal representative is willing to allow his or her primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial, if the primary care practitioner/consultant is different to the investigator.

You may not qualify if:

• Participant has clinically significant unstable medical conditions other than epilepsy.
• Participant experiences \> 300 total seizures within the first 28 days of the baseline period.
• Participant has any prior exposure to GWP42003-P.
• Participant has initiated felbamate within the last 12 months.
• Participant has initiated mammalian target of rapamycin (mTOR) inhibitors for epilepsy within the last 4 weeks.
• Participant is currently using or has in the past used recreational or medicinal cannabis or synthetic cannabinoid-based medications (including Sativex®) within the 3 months prior to trial entry.
• Participant has had clinically relevant symptoms or a clinically significant illness, other than epilepsy, in the 4 weeks prior to screening or Visit 2.
• Participant has laboratory values at screening or Visit 2 that are clinically significantly abnormal in the investigator's opinion.
• Participant tests positive for Δ9-tetrahydrocannabinol (THC) or cannabidiol (CBD) at screening.
• Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of GWP42003-P.
• Participant has significantly impaired hepatic function at the screening visit, defined as any of the following:
• Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 5 × upper limit of normal (ULN);
• Serum ALT or AST \> 3 × ULN and (total bilirubin \[TBL\] \> 2 × ULN or international normalized ratio \[INR\] \> 1.5);
• Serum ALT or AST \> 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (\> 5%).
• Participant has received an investigational medical product within the 3 months prior to the screening visit.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

MeSH Terms

Conditions

Lennox Gastaut Syndrome

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Epileptic Syndromes; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2019
• First Posted: October 21, 2019
• Study Start: October 1, 2020
• Primary Completion: December 1, 2021
• Study Completion: December 1, 2021
• Last Updated: September 1, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share